How I approach disease-modifying therapy in children with sickle cell disease in an era of novel therapies.
Pediatr Blood Cancer
; 68(12): e29363, 2021 12.
Article
em En
| MEDLINE
| ID: mdl-34550643
ABSTRACT
Finally,after decades of stagnation, the therapeutic landscape for sickle cell disease (SCD) is changing with an increasing number of novel therapeutics. Hydroxyurea remains the primary disease-modifying therapy and, when started early in life with maintenance of an optimal dose, can reduce many SCD-related complications. To complement hydroxyurea, there are a growing number of pharmacologic options with additional efforts focused on the development and optimization of curative therapies. Here, we review current treatment options and provide recommendations as to how to approach the treatment of children and adolescents within this evolving therapeutic landscape to allow for full and healthy lives.
Palavras-chave
Texto completo:
1
Bases de dados:
MEDLINE
Assunto principal:
Hidroxiureia
/
Anemia Falciforme
Tipo de estudo:
Guideline
Limite:
Adolescent
/
Child
/
Humans
Idioma:
En
Revista:
Pediatr Blood Cancer
Assunto da revista:
HEMATOLOGIA
/
NEOPLASIAS
/
PEDIATRIA
Ano de publicação:
2021
Tipo de documento:
Article
País de afiliação:
Estados Unidos